Efficient generation of retinal photoreceptors from stem/progenitor cells.

Unmet Need
Approximately 253 million people worldwide live with vision impairment. Of those with vision impairment, approximately 15% are blind. Most human blindness is caused by the dysfunction or death of retinal photoreceptors. For many eye disorders, there is no way to reverse photoreceptor loss, and efforts to treat vision loss are aimed at slowing or preventing the disease. Current methods to produce retinal photoreceptors are challenging, laborious and limit research efforts into understanding the mechanisms of photoreceptor death. Consequently, a better method of generating retinal photoreceptors is needed to improve research efforts and develop therapies for restoration of vision in affected individuals.  
 
Technology Overview
The inventors have identified an approach to robustly generate pure populations of retinal photoreceptors. The inventors selectively inactivating a family of transcription factors: Nfia, Nfib, and Nfix, individually and in combination, in the retina of mice. This inactivation led late-stage retinal progenitors to proliferate into rod photoreceptors and additional progenitors. Thus, the inventors were able to produce a high yield population of retinal photoreceptors from retinal progenitor cells (RPCs). Importantly, the inventors used single cell RNA-sequencing to compare these cell culture-produced retinal photoreceptors to normal rods, and found they had similar expression profiles.
 
Stage of Development
The inventors have tested mice with Nfia, Nfib, and Nfix all inactivated in the retina and found that this led to the development of pure rod photoreceptor populations, with similar characteristics to normal rods.
 
Publications
Clark, B et al. BioRxiv. 2018 30 July, 378950.
 

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